Ultragenyx Pharmaceutical Company Description
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific.
Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia.
The company’s products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome.
It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III.
Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
| Country | United States |
| Founded | 2010 |
| Industry | Biological Products, Except Diagnostic Substances |
| Employees | 1,294 |
| CEO | Emil Kakkis |
Contact Details
Address: 60 Leveroni Court Novato, Delaware 94949 United States | |
| Phone | 415 483 8800 |
| Website | ultragenyx.com |
Stock Details
| Ticker Symbol | UP0 |
| Exchange | Frankfurt Stock Exchange |
| Stock Type | Common Stock |
| Fiscal Year | January - December |
| Reporting Currency | USD |
| SIC Code | 2836 |
Key Executives
| Name | Position |
|---|---|
| Emil Kakkis | Chief Executive Officer |
| Howard Horn | Chief Financial Officer |